Ionis Completes Enrollment in Pivotal Phase 3 REVEAL Angelman Syndrome Study
IONS•Ionis Pharmaceuticals has completed enrollment of the pivotal cohort in its Phase 3 REVEAL study evaluating obudanersen for Angelman syndrome. This milestone clears the way for primary efficacy assessments and positions obudanersen for a potential regulatory submission upon topline data readout.
1. Enrollment Milestone
Ionis Pharmaceuticals has announced full enrollment of the pivotal treatment cohort in its Phase 3 REVEAL trial of obudanersen in Angelman syndrome, marking a critical step toward evaluating the therapy’s efficacy and safety in the target population.
2. Trial Design and Objectives
The REVEAL study is a randomized, double-blind, placebo-controlled trial designed to assess obudanersen’s impact on neurodevelopmental and clinical outcomes in patients with Angelman syndrome, with primary endpoints centered on motor function, communication abilities and seizure frequency over a 52-week treatment period.
3. Next Steps and Timelines
Following cohort enrollment, Ionis will complete safety reviews and initiate primary efficacy analyses; topline results are anticipated after database lock, which will inform potential submission of a new drug application for obudanersen.
